Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03172455
• Date of registration: 29/05/2017
• Prospective Registration: No
• Primary sponsor: Impatients N.V. trading as myTomorrows
• Public title: Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)
• Scientific title: Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)
• Date of first enrolment: May 1, 2017
• Recruitment status: No longer available
• URL: https://clinicaltrials.gov/show/NCT03172455
• Study type: Expanded Access

Key inclusion & exclusion criteria

Inclusion Criteria:

- This program is available for patients who suffer from steroid refractory acute GvHD
after HSCT, or for whom, in the opinion of their treating physician, other treatment
options or clinical trials in this indication are unsuitable.

- The disease indication for which the participant required HSCT must be in remission

- Acute graft-versus-host disease (aGvHD), including lower GI involvement (modified

- International Bone Marrow Transplant Registry (IBMTR) Severity Stage 1 to 4 [>500 mL
diarrhea/day]), with or without other organ system involvement.

- For women of childbearing potential, had a negative serum or urine pregnancy test
within 14 days prior to enrolment.

Exclusion Criteria:

- Participant with manifestations of chronic GvHD

- Participant with acute/chronic GvHD overlap syndrome

- Participant whose GvHD developed after donor lymphocyte infusion

- Participant with severe sepsis involving at least 1 organ failure

- Participant who is seropositive or positive in the nucleic acid test for human
immunodeficiency virus (HIV)

- Participant with active hepatitis B or C

- If female, participant is pregnant or lactating at the time of enrollment, or has
plans to become pregnant during the program



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Alpha-1 Protease Inhibitor Deficiency

Graft Vs Host Disease

Steroid Refractory Acute Graft Versus Host Disease

Alpha-1 Proteinase Inhibitor

Graft-versus-host-disease

Acute Graft-Versus-Host Reaction Following Bone Marrow Transplant

Acute-graft-versus-host Disease

Alpha 1-Antitrypsin Deficiency

Intervention(s)

Drug: Glassia

Secondary ID(s)

2017-AATGLAKAM-EU

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Kamada, Ltd.